Idebenone- a promising drug for Friedreich's ataxia
Friedreich's ataxia (FRDA) is considered a rare severe disease of slow progression that leads to a loss of ambulation after 5 to 10 years from its onset. Some symptoms include scoliosis, leg malformations and very often cardiomyopathy, where patients undergo physiotherapy and sometimes surgery. Most importantly, physiological support to the patients and their families is very important since FDRA patients are frequently bed ridden with a very bad quality of life. Until 1999, no effective drug for FRDA was found, when a new compound called idebenone displayed increased efficacy in a cellular system coming from human heart tissue. As a continuation of this demonstration, this project focused on further, more advanced studies on the efficacy of idebenone as a treatment of FRDA cardiomyopathy. Therefore, they employed intact cells that are deficient in or lacking frataxin -the responsible mitochondrial protein, taken from transgenic mice models and human samples for preclinical tests. The product's efficiency was established on the three different systems offering some interesting results. More specifically, idebenone was found to be a robust antioxidant with a great potential to defend different oxidative attacks in situ in living cells. In addition, it may antagonise heart hypertrophy in mice models and patients, using a different approach. Moreover, sufficient residual frataxin coupled with idebebone administration may result in the restoration of iron-sulphur cluster-containing protein activity, whose loss is specifically observed in FRDA patients. Collaboration is sought for the development/distribution of the novel antioxidant molecules to control FRDA disease.
